XT-150-1-0402

About this trial

Last updated 6 months ago

Study ID

XT-150-1-0402

Status

Not yet recruiting

Type

Interventional

Phase

Phase 1

Placebo

Accepting

18 to 80 Years

All

Not accepting

Healthy Volunteers

Trial Timing

Started 4 months ago

This is a Phase 1, open-label, multi-center safety study of XT-150 in adult participants with Amyotrophic Lateral Sclerosis (ALS). Participants providing informed consent and meeting all study eligibility criteria will be enrolled in the study and will receive a single injection of XT-150 at the Baseline visit. Follow-up visits will occur over 180 days (6 months) after the injection. 8 participants (4 participants per dose level) will be enrolled sequentially in up to 2 ascending, single dose cohorts: Cohort 1: 1.5 mg XT-150 Cohort 2: 4.5 mg XT-150

Key Inclusion Criteria:

- Adults between 18 and 80 years of age

- Male or female, if of childbearing potential or sexually active, strict
contraception required

- Have ALS diagnosed by a doctor (specifically, sporadic or familial ALS diagnosed as
clinically probable, lab-supported probable or definite ALS defined by the El
Escorial criteria)

- Have had symptoms of ALS (muscle weakness) within 36 months of starting this study

- Have the ability to slowly exhale a volume of air at least 60% of what is expected
for the participant's sex, height and age

- Have not received treatment for ALS or are currently on a stable dose of an approved
treatment for ALS. Patients currently receiving Tofersen are not eligible.

- Able to receive the study injection intrathecally, determined by the study doctor

- Able to undergo the study procedures and adhere to the study visit schedule at the
time of study entry, with an estimated life expectancy of 6 months or greater

Key Exclusion Criteria:

- Have an implanted shunt to drain cerebrospinal fluid (CSF) or an implanted CNS
catheter

- Have an implanted of diaphragm pacing system

- Tracheostomy

- History or current diagnosis of cardiac conditions or ECG abnormalities indicating
significant risk of safety for participants in the study

- History or current diagnosis of respiratory conditions such as COPD

- History or current diagnosis of cancer, chemical meningitis, HIV, Hep B, Hep C,
uncontrolled diabetes

- Presence of an autoimmune condition (for example, rheumatoid arthritis or lupus)
requiring treatment or immunodeficiency

- Clinical or laboratory evidence of hepatic or renal disease/injury.

- Taking any prohibited medications

- Women who are pregnant or nursing

- Use of any investigational drugs or devices within 30 days or 5 half-lives of the
study agent (whichever is longer). Exception: Observational, non-interventional
clinical studies are allowed in the opinion of the study doctor.

- Any other condition that the study doctor feels could compromise the participant's
safety, ability to communicate with the study staff, or the quality of the data

Safety Study of XT-150 in Participants with ALS